European regulators have given PTC Therapeutics (PTCT) a five-year regulatory reprieve on its Duchenne muscular dystrophy drug Translarna, sending the company's stock price soaring by more than 80% in premarket trading Friday morning.
The conditional approval of Translarna in Europe should be renewed, conditioned on PTC Therapeutics conducting another randomized, placebo-controlled study, according to the recommendation released Friday by a committee of drug reviewers from the European Medicines Agency.
The proposed study of Translarna in Duchenne muscular dystrophy patients will take five years to conduct, but in what is great news for PTC Therapeutics, the company will be allowed to sell the drug during that period.
For that reason, PTC Therapeutics shares nearly doubled in price to $11.80 in Friday premarket trading.
Translarna is designed to treat Duchenne caused by a so-called "nonsense" mutation. The drug targets a different set of Duchenne patients than those treated by Sarepta Therapeutics' (SRPT) newly approved Exondys.
PTC Therapeutics secured conditional approval in Europe based on a phase III study which failed to demonstrate a conclusive benefit for Duchenne patients. Most recently, the company had warned investors that European regulators were leaning towards not renewing Translarna's conditional approval, so Friday's decision came as a surprise.
"Although the data available to date continue to indicate that Translarna slows the progression of the disease and there are no major safety concerns, the Committee considered that further comprehensive data are still needed to fully confirm that the benefit-risk balance of the medicine is positive," European regulators said Friday.
The willingness of European regulators to be flexible with Translarna should also bode well for Sarepta as it moves forward with plans to seek marketing clearance there.